In Their Lifetime: Outlook for Rare Disease and Orphan Drug Research

Collectively rare diseases comprise a large segment of the global population. Developing effective treatments to combat these diseases demand heavy resources from biopharmaceutical companies - both monetary and scientific. Even though these diseases are singularly rare, the combination of governmental incentives to develop compounds for potential success, combined with public support to find cures for these diseases make the practice of R&D for Rare Diseases-Orphan Drug one of the fastest growing segments in the biopharmaceutical industry.

Spotlight

Anacor Pharmaceuticals

Anacor is a biopharmaceutical company focused on discovering, developing and commercializing novel small-molecule therapeutics derived from its boron chemistry platform. Anacor has discovered and is developing five clinical compounds, including its three lead programs: AN2690, a topical antifungal for the treatment of onychomycosis; AN2728, a topical anti-inflammatory PDE-4 inhibitor for the treatment of psoriasis; and GSK 2251052, or GSK '052 (formerly referred to as AN3365), a systemic antibiotic for the treatment of infections caused by Gram-negative bacteria, which has been licensed to GlaxoSmithKline under the companies' research and development agreement.

OTHER WHITEPAPERS
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whitePaper | November 1, 2022

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whitePaper | August 8, 2022

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The expansion in the development and manufacturing of highly potent active pharmaceutical ingredients (HPAPIs) and complex active pharmaceutical ingredients (APIs) has grown tremendously as demands in healthcare to bring newer therapies to market quicker increase.

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whitePaper | January 5, 2023

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Spotlight

Anacor Pharmaceuticals

Anacor is a biopharmaceutical company focused on discovering, developing and commercializing novel small-molecule therapeutics derived from its boron chemistry platform. Anacor has discovered and is developing five clinical compounds, including its three lead programs: AN2690, a topical antifungal for the treatment of onychomycosis; AN2728, a topical anti-inflammatory PDE-4 inhibitor for the treatment of psoriasis; and GSK 2251052, or GSK '052 (formerly referred to as AN3365), a systemic antibiotic for the treatment of infections caused by Gram-negative bacteria, which has been licensed to GlaxoSmithKline under the companies' research and development agreement.

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